Allon Therapeutics Inc. announced that it has reached agreement with the United States Food and Drug Administration (FDA) on a Special Protocol Assessment (SPA) for a pivotal Phase 2/3 clinical trial to evaluate the Company’s lead neuroprotective drug candidate, davunetide, as a potential treatment for progressive supranuclear palsy (PSP), a rapidly-progressing and fatal degenerative brain disease.
The SPA is a mechanism through which the FDA and Allon reach agreement on the design, size, clinical endpoints, and data analysis of a clinical trial that is intended to support an efficacy claim in a New Drug Application (NDA) for regulatory approval. The SPA ensures that the agreed clinical trial design meets the FDA’s expectations for a pivotal study. Allon believes that if this pivotal study generates statistically significant and consistent data, davunetide will be considered by FDA for approval in PSP.
Gordon McCauley, President and CEO of Allon, said the SPA agreement gives Allon a clear path to develop davunetide for the treatment of PSP and provides a foundation for further development in other brain diseases involving tau pathology.
“This SPA is an important part of our regulatory strategy for davunetide. We believe the pivotal trial agreed to in the SPA will generate data in about 2 years that will form the basis of approval of davunetide in PSP,” said McCauley.
“Most important is the opportunity to help thousands of patients who suffer from a debilitating and fatal disease,” said McCauley. “PSP patients and their families endure an unspeakable tragedy, often dying a little more than three years after diagnosis.”
“Our research to date suggests that davunetide will work in patients with the tau pathology which is a characteristic feature of PSP. Given these outcomes and that there is a recognized rating scale measuring clinically relevant outcomes, we believe there is a strong rationale to pursue approval in PSP.”
The pivotal Phase 2/3 randomized, double-blinded, placebo controlled trial in PSP will enroll 300 patients in the United States, Canada, United Kingdom, France, Germany, and Australia. Study participants will receive either placebo or 30 mg of davunetide administered twice daily for a period of 12-months.
The primary outcome measures will be the Progressive Supranuclear Palsy Rating Scale (PSPRS) and the Schwab and England Activities of Daily Living (SEADL) scale. Secondary measures will include Clinical Global Impression (CGI) and brain imaging by magnetic resonance tomography (MRI). Additional exploratory endpoints include cognitive and executive function as well as cerebrospinal fluid (CSF) biomarkers.
Allon previously announced that davunetide received orphan drug designation in the United States (January 12, 2010) and the European Union (March 17, 2010), Fast Track Status with the FDA (April 6, 2010), and that davunetide met the primary outcome measure of safety and tolerability, with potential trends from secondary efficacy endpoints, in a pilot study of 12 patients suffering PSP and related tau pathology disorders (October 8, 2010).
Date: January 5, 2010
Source: Allon Therapeutics Inc.
Filed Under: Drug Discovery